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WO2011125054A3 - Protein and nucleic acid delivery vehicles, components and mechanisms thereof - Google Patents

Protein and nucleic acid delivery vehicles, components and mechanisms thereof Download PDF

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Publication number
WO2011125054A3
WO2011125054A3 PCT/IB2011/051533 IB2011051533W WO2011125054A3 WO 2011125054 A3 WO2011125054 A3 WO 2011125054A3 IB 2011051533 W IB2011051533 W IB 2011051533W WO 2011125054 A3 WO2011125054 A3 WO 2011125054A3
Authority
WO
WIPO (PCT)
Prior art keywords
dna
phage
packaging
delivery vehicles
proheads
Prior art date
Application number
PCT/IB2011/051533
Other languages
French (fr)
Other versions
WO2011125054A2 (en
WO2011125054A4 (en
Inventor
Venigalla Rao
Original Assignee
The Catholic University Of America
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by The Catholic University Of America filed Critical The Catholic University Of America
Priority to EP11765160.4A priority Critical patent/EP2542681B1/en
Priority to CN201180030396.9A priority patent/CN103003431B/en
Priority to JP2013503215A priority patent/JP5863766B2/en
Publication of WO2011125054A2 publication Critical patent/WO2011125054A2/en
Publication of WO2011125054A3 publication Critical patent/WO2011125054A3/en
Publication of WO2011125054A4 publication Critical patent/WO2011125054A4/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6901—Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2795/00—Bacteriophages
    • C12N2795/00011—Details
    • C12N2795/10011—Details dsDNA Bacteriophages
    • C12N2795/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2795/10042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2795/00—Bacteriophages
    • C12N2795/00011—Details
    • C12N2795/10011—Details dsDNA Bacteriophages
    • C12N2795/10111—Myoviridae
    • C12N2795/10122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2795/00—Bacteriophages
    • C12N2795/00011—Details
    • C12N2795/10011—Details dsDNA Bacteriophages
    • C12N2795/10111—Myoviridae
    • C12N2795/10141—Use of virus, viral particle or viral elements as a vector
    • C12N2795/10142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2795/00—Bacteriophages
    • C12N2795/00011—Details
    • C12N2795/10011—Details dsDNA Bacteriophages
    • C12N2795/10111—Myoviridae
    • C12N2795/10151—Methods of production or purification of viral material
    • C12N2795/10152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Complex viruses are assembled from simple protein subunits by sequential and irreversible assembly. During genome packaging in bacteriophages, a powerful molecular motor assembles at the special portal vertex of an empty prohead to initiate packaging. An aspect of the invention relates to the phage T4 packaging machine being highly promiscuous, translocating DNA into finished phage heads as well as into proheads. Single motors can force exogenous DNA into phage heads at the same rate as into proheads and phage heads undergo repeated initiations, packaging multiple DNA molecules into the same head. This shows that the phage DNA packaging machine has unusual conformational plasticity, powering DNA into an apparently passive capsid receptacle, including the highly stable virus shell, until it is full. These features allow for the design of a novel class of nanocapsid delivery vehicles.
PCT/IB2011/051533 2010-04-09 2011-04-08 Protein and nucleic acid delivery vehicles, components and mechanisms thereof WO2011125054A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
EP11765160.4A EP2542681B1 (en) 2010-04-09 2011-04-08 Protein and nucleic acid delivery vehicles, components and mechanisms thereof
CN201180030396.9A CN103003431B (en) 2010-04-09 2011-04-08 Protein and nucleic acid delivery vehicles, their components and their mechanisms
JP2013503215A JP5863766B2 (en) 2010-04-09 2011-04-08 Protein and nucleic acid delivery vehicles, components and mechanisms

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US32233410P 2010-04-09 2010-04-09
US61/322,334 2010-04-09
US13/082,466 US8802418B2 (en) 2010-04-09 2011-04-08 Protein and nucleic acid delivery vehicles, components and mechanisms thereof
US13/082,466 2011-04-08

Publications (3)

Publication Number Publication Date
WO2011125054A2 WO2011125054A2 (en) 2011-10-13
WO2011125054A3 true WO2011125054A3 (en) 2012-05-10
WO2011125054A4 WO2011125054A4 (en) 2012-06-28

Family

ID=44761088

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2011/051533 WO2011125054A2 (en) 2010-04-09 2011-04-08 Protein and nucleic acid delivery vehicles, components and mechanisms thereof

Country Status (5)

Country Link
US (3) US8802418B2 (en)
EP (2) EP2703491B1 (en)
JP (2) JP5863766B2 (en)
CN (2) CN103725703B (en)
WO (1) WO2011125054A2 (en)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
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AU2004299139A1 (en) * 2003-12-17 2005-06-30 The Catholic University Of America Methods and compositions comprising bacteriophage nanoparticles
US9163262B2 (en) 2003-12-17 2015-10-20 The Catholic University Of America In vitro and in vivo delivery of genes and proteins using the bacteriophage T4 DNA packaging machine
US20120214867A1 (en) * 2011-02-18 2012-08-23 The Government Of The United States Of America, As Represented By The Secretary Of The Navy Bacteriophage T4 Nanoparticles for Eukaryotic Delivery
US9765122B2 (en) 2013-01-18 2017-09-19 University Of Washington Through Its Center For Commercialization Theragnostic particles
US9328149B2 (en) 2013-07-12 2016-05-03 The Catholic University Of America Mutated and bacteriophage T4 nanoparticle arrayed F1-V immunogens from Yersinia pestis as next generation plague vaccines
KR102423377B1 (en) 2013-08-05 2022-07-25 트위스트 바이오사이언스 코포레이션 De novo synthesized gene libraries
WO2016115522A2 (en) * 2015-01-16 2016-07-21 University Of Kentucky Research Foundation Lipid bilayer-integrated spp1 connector protein nanopore and spp1 connector protein variants for use as lipid bilayer-integrated nanopore
US10669304B2 (en) 2015-02-04 2020-06-02 Twist Bioscience Corporation Methods and devices for de novo oligonucleic acid assembly
US9981239B2 (en) 2015-04-21 2018-05-29 Twist Bioscience Corporation Devices and methods for oligonucleic acid library synthesis
CA2998169A1 (en) 2015-09-18 2017-03-23 Twist Bioscience Corporation Oligonucleic acid variant libraries and synthesis thereof
KR102794025B1 (en) 2015-09-22 2025-04-09 트위스트 바이오사이언스 코포레이션 Flexible substrates for nucleic acid synthesis
WO2017160752A1 (en) 2016-03-14 2017-09-21 Intellia Therapeutics, Inc. Methods and compositions for gene editing
WO2017165859A1 (en) * 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
US10417457B2 (en) 2016-09-21 2019-09-17 Twist Bioscience Corporation Nucleic acid based data storage
CN110892485B (en) 2017-02-22 2024-03-22 特韦斯特生物科学公司 Nucleic acid-based data storage
CN106939355A (en) * 2017-03-01 2017-07-11 苏州系统医学研究所 A kind of screening of influenza virus attenuated live vaccines strain and authentication method
CN108949701A (en) * 2017-05-26 2018-12-07 中国科学院深圳先进技术研究院 A kind of the virus injection method and application of monkey class brain area
WO2018226602A1 (en) 2017-06-05 2018-12-13 Research Institute At Nationwide Children's Hospital Enhanced modified viral capsid proteins
KR102637566B1 (en) 2017-10-20 2024-02-16 트위스트 바이오사이언스 코포레이션 Heated nanowells for polynucleotide synthesis
EP3814497A4 (en) 2018-05-18 2022-03-02 Twist Bioscience Corporation POLYNUCLEOTIDES, REAGENTS, AND METHODS FOR NUCLEIC ACID HYBRIDIZATION
US11827877B2 (en) 2018-06-28 2023-11-28 Crispr Therapeutics Ag Compositions and methods for genomic editing by insertion of donor polynucleotides
CN119708086A (en) 2018-12-26 2025-03-28 特韦斯特生物科学公司 Highly accurate de novo polynucleotide synthesis
US11492727B2 (en) 2019-02-26 2022-11-08 Twist Bioscience Corporation Variant nucleic acid libraries for GLP1 receptor
CN113785057A (en) 2019-02-26 2021-12-10 特韦斯特生物科学公司 Variant nucleic acid libraries for antibody optimization
US20210047649A1 (en) 2019-05-08 2021-02-18 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
EP3983545A1 (en) 2019-06-17 2022-04-20 Vertex Pharmaceuticals Incorporated Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
US11155835B2 (en) * 2019-08-19 2021-10-26 The Catholic University Of America Prokaryotic-eukaryotic hybrid viral vector for delivery of large cargos of genes and proteins into human cells
WO2021061829A1 (en) 2019-09-23 2021-04-01 Twist Bioscience Corporation Variant nucleic acid libraries for crth2
WO2021061842A1 (en) 2019-09-23 2021-04-01 Twist Bioscience Corporation Variant nucleic acid libraries for single domain antibodies
WO2021140615A1 (en) * 2020-01-09 2021-07-15 哲朗 近藤 Novel recombinant bacteriophage

Citations (2)

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US20080274533A1 (en) * 2007-03-01 2008-11-06 The Catholic University Of America T4 bacteriophage bound to a substrate

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US20080274533A1 (en) * 2007-03-01 2008-11-06 The Catholic University Of America T4 bacteriophage bound to a substrate

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PRISCILLA KEMP ET AL.: "Changes in bacteriophage T7 virion structure at the initiation of infection", VIROLOGY, vol. 340, no. 2, 2005, pages 307 - 317, XP005078433 *
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Also Published As

Publication number Publication date
EP2542681A4 (en) 2013-10-16
EP2703491A1 (en) 2014-03-05
EP2542681B1 (en) 2019-02-27
JP5863766B2 (en) 2016-02-17
JP2013526850A (en) 2013-06-27
CN103003431B (en) 2014-11-26
CN103003431A (en) 2013-03-27
WO2011125054A2 (en) 2011-10-13
EP2542681A2 (en) 2013-01-09
EP2703491B1 (en) 2017-03-08
US8802418B2 (en) 2014-08-12
WO2011125054A4 (en) 2012-06-28
CN103725703B (en) 2016-02-10
US20140335158A1 (en) 2014-11-13
US20110250263A1 (en) 2011-10-13
JP6127124B2 (en) 2017-05-10
CN103725703A (en) 2014-04-16
US20130196416A1 (en) 2013-08-01
JP2016093183A (en) 2016-05-26
US9523101B2 (en) 2016-12-20
US9365867B2 (en) 2016-06-14

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